Cornelis Korstiaan van der Ent

A functional comparison of readthrough agent ELX-02 across a wide range of nonsense CFTR variants

This study evaluates ELX-02's effectiveness in restoring CFTR function across various nonsense variants using patient-derived intestinal organoids, finding limited and variable rescue effects.

Personalized multi-assay profiling of respiratory motile ciliopathies and mRNA therapy

This study develops a personalized multi-assay platform using nasal epithelial cells to assess ciliary function and therapeutic response in primary ciliary dyskinesia.

Small molecule-directed differentiation of submerged-cultured human nasal airway epithelia for respiratory disease modelling

This study develops a method to differentiate human nasal epithelial cells into airway-like tissues using small molecules, creating disease models for respiratory conditions.

FDA-Approved Drug Screening in Patient-Derived Organoids Demonstrates Potential of Drug Repurposing for Rare Cystic Fibrosis Genotypes

The paper screens 1400 FDA-approved drugs in CF patient-derived intestinal organoids using a functional assay to identify potential repurposing for rare CF genotypes.

Infant RSV immunoprophylaxis changes nasal epithelial DNA methylation at six years of age

This study shows that infant RSV immunoprophylaxis with palivizumab alters nasal epithelial DNA methylation at age 6, affecting antiviral defense pathways but not asthma.